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November 9, 2020

Reforming the Process for Deep Brain Stimulation and Neurologic Device Approval in Rare Diseases

Author Affiliations
  • 1Norman Fixel Institute for Neurological Diseases, University of Florida Health, Gainesville
  • 2Associate Editor, JAMA Neurology
  • 3Neuroethics Studies Program, Pellegrino Center for Clinical Bioethics, Department of Neurology, Georgetown University Medical Center, Washington, DC
JAMA Neurol. 2021;78(1):5-6. doi:10.1001/jamaneurol.2020.4232

Traditionally, we have developed and offered neurologic therapies for the “many” (ie, individuals with epidemiologically more prevalent disorders), but the possibility exists to serve “the few” who have more infrequently occurring diseases and therefore represent smaller patient populations. The US Orphan Drug Act of 1983 defined a rare disease or condition as any pathology affecting fewer than 200 000 people. Pharmaceutical companies can request an orphan drug designation for the use of specific drugs and/or biologics in treating rare conditions. Incentives for obtaining this status include tax credits for human clinical trials, access to an Orphan Product Grants Program, waiver of the prescription drug user fee of $3 million, and market exclusivity for 7 years.1 Unfortunately, the process for neurotechnological devices that are developed and/or can be used for rare diseases or conditions does not entail, or benefit from, the same approval process.

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    1 Comment for this article
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    Deep Brain Stimulation and other Neurostimulation or Neuromodulatory Devices
    Khichar Shubhakaran, MD (Med), D.M.(Neurology) | Senior Professor and Head of department of Neurology, M D M Hospital, Dr. S. N. Medical College, Jodhpur (Rajasthan), India-342003
    The brain or nervous stimulatory or neuromodulatory devices are a little bit novel and are intended to improve the efficacy in treatment of untreatable or refractory cases. The cost and approval is a big hurdle in development and application of these devices. Here the concerned government may subsidize the cost so as to make them more cost effective. These devices are quite complicated technically so they need to be better tailored in order to aid clinicians with technical support. It may be worthwhile to incorporate a potential provision of a separate operator's interpretation. This may of course also increase the cost, but such things may be quite beneficial in long term use. These two modalities may be compared later and in case of any discrepancy the device may be modified or corrected accordingly.
    CONFLICT OF INTEREST: None Reported
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